Characterizing and Quantifying Impacts of Neuromyelitis Optica Spectrum Disorder (NMOSD) on Patients and Caregivers
Author(s)
Benjamin J. Osborne, MD1, Mayvis Rebeira, PhD2, Roc Reguant, PhD3, Jesus Loreto, AS4, Jamie Sullivan, MPH5, Elizabeth Crevier, MPH6, Ashlee Olsen, Patient Consultant7, Evanthia Bernitsas, MD8.
1Medstar Georgetown University Medical Center, Washington, DC, USA, 2Alexion, AstraZeneca Rare Disease (affiliation at the time research was conducted), Mississauga, ON, Canada, 3Alexion, AstraZeneca Rare Disease, Barcelona, Spain, 4Caregiver Consultant, Unspecified, FL, USA, 5EveryLife Foundation for Rare Diseases, Washington, DC, USA, 6Avalere Health, Washington, DC, USA, 7Patient Consultant, Orlando, FL, USA, 8Creighton University School of Medicine, Omaha, NE, USA.
1Medstar Georgetown University Medical Center, Washington, DC, USA, 2Alexion, AstraZeneca Rare Disease (affiliation at the time research was conducted), Mississauga, ON, Canada, 3Alexion, AstraZeneca Rare Disease, Barcelona, Spain, 4Caregiver Consultant, Unspecified, FL, USA, 5EveryLife Foundation for Rare Diseases, Washington, DC, USA, 6Avalere Health, Washington, DC, USA, 7Patient Consultant, Orlando, FL, USA, 8Creighton University School of Medicine, Omaha, NE, USA.
OBJECTIVES: The impact of rare diseases, such as NMOSD, encompasses areas of daily life that are not captured by traditional analyses. The true total burden of the disease extends beyond direct excess medical costs. This survey captured which domains were impacted from the perspective of both patients with NMOSD and caregivers of patients with NMOSD.
METHODS: Prior research identified 25 patient-centered “impact elements” across 8 life domains (occupational, financial, emotional health, physical health, sleep, social, planning and autonomy, and safety). Patients and unpaired caregivers independently recruited through an external firm and patient/caregiver networks completed a subsequent electronic, quantitative survey (January-April 2024; available in English and Spanish), rating the 25 impact elements on relevance and severity (1 [mild] to 5 [severely limiting]).
RESULTS: Respondents included 209 patients with NMOSD (75% aged 18-54y; 76% female) and 49 caregivers (82% aged 18-54y; 57% female). The top 3 domains most impacted by NMOSD for patients were occupational (average domain severity score [SD]: 3.8 [1.2]), physical health (3.7 [1.2]), and planning and autonomy (3.7 [1.2]). For caregivers, the top 3 domains most impacted were sleep (3.6 [1.2]), planning and autonomy (3.6 [1.0]), and occupational (3.6 [1.2]). Among patients, the elements with highest severity impact were downstream health effects; personal/professional development; and hobbies, leisure, or volunteering. Patients with ≥1 relapse in 2023 reported higher severity across 76% (19/25) of impact elements compared with patients without relapse, including feelings of sadness and hopelessness; and anger, frustration, or fear. Among caregivers, the elements with highest severity impact were disruption to occupational advancement and disruption to future planning.
CONCLUSIONS: This study quantified the impacts experienced by US patients with NMOSD and caregivers, which extend beyond symptoms and direct excess medical costs. These findings emphasize the importance of measuring perspectives from both patients and caregivers to fully understand the burden of NMOSD.
METHODS: Prior research identified 25 patient-centered “impact elements” across 8 life domains (occupational, financial, emotional health, physical health, sleep, social, planning and autonomy, and safety). Patients and unpaired caregivers independently recruited through an external firm and patient/caregiver networks completed a subsequent electronic, quantitative survey (January-April 2024; available in English and Spanish), rating the 25 impact elements on relevance and severity (1 [mild] to 5 [severely limiting]).
RESULTS: Respondents included 209 patients with NMOSD (75% aged 18-54y; 76% female) and 49 caregivers (82% aged 18-54y; 57% female). The top 3 domains most impacted by NMOSD for patients were occupational (average domain severity score [SD]: 3.8 [1.2]), physical health (3.7 [1.2]), and planning and autonomy (3.7 [1.2]). For caregivers, the top 3 domains most impacted were sleep (3.6 [1.2]), planning and autonomy (3.6 [1.0]), and occupational (3.6 [1.2]). Among patients, the elements with highest severity impact were downstream health effects; personal/professional development; and hobbies, leisure, or volunteering. Patients with ≥1 relapse in 2023 reported higher severity across 76% (19/25) of impact elements compared with patients without relapse, including feelings of sadness and hopelessness; and anger, frustration, or fear. Among caregivers, the elements with highest severity impact were disruption to occupational advancement and disruption to future planning.
CONCLUSIONS: This study quantified the impacts experienced by US patients with NMOSD and caregivers, which extend beyond symptoms and direct excess medical costs. These findings emphasize the importance of measuring perspectives from both patients and caregivers to fully understand the burden of NMOSD.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR43
Topic
Patient-Centered Research
Topic Subcategory
Patient Engagement, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Neurological Disorders, Rare & Orphan Diseases